Pediatric Preclinical Testing Consortium: Research Programs non-CNS

GCCRI的研究小组将继续进行研究，重点是确定活性药物， 使用儿科肉瘤和肾肿瘤的异种移植物模型的组合。我们将完成 正在进行的研究，并开始新的研究，如下所述。此外，我们计划描述 18个新的肉瘤PDX模型（外显子组测序和RNAseq），以及33个完整的表征 PDX模型中，我们有来自TARGET计划的外显子组序列数据，但我们 缺乏RNAseq数据。这将极大地扩大可用于肿瘤研究的肉瘤模型的数量。 FNIH正在开发的未来PPP中使用。我们延长6个月的目标是： 具体目标 延长期内拟议的研究。 1.使用Amgen 509（ES-7、ES-3、EW-5）完成研究。 2.完成ONC 201的体内研究。 3.对PPTC药物/组合进行筛查。 4.在肉瘤和肾脏模型中评价PLX-038 A联合长春新碱。 5.评价Talazoparib +辐射在体外和体内CHLA 258细胞中的作用，然后在合理的情况下进行扩增 6.通过RNAseq表征ALS队列中遗漏的33个模型。 7表征18个横纹肌肉瘤和5个尤文肉瘤PDX模型，之前没有 表征了 8.完成报告PPTC结果的手稿的提交。

The group at GCCRI will continue studies focused on identifying active agents and combinations using xenograft models of pediatric sarcoma and kidney tumors. We will complete ongoing studies, and start new studies as described below. In addition we plan to characterize 18 new sarcoma PDX models (exome seq and RNAseq), and complete characterization of 33 PDX models where we have exome sequence data from the TARGET initiative, but where we lack RNAseq data. This will greatly expand the number of available sarcoma models that can be used in the future PPP being developed by the FNIH. Our aims for the 6 month extension are: Specific Aims Proposed studies during the extension period. 1. Complete studies with Amgen 509 (ES-7, ES-3, EW-5). 2. Complete in vivo studies with ONC201. 3. Undertake screening for PPTC drugs/combinations. 4. Evaluate PLX-038A combined with vincristine in sarcoma and kidney models. 5. Evaluate Talazoparib + Radiation in CHLA258 cell in vitro and in vivo, then expand if justified 6. To characterize 33 models by RNAseq that were omitted from the ALS cohort. 7 To characterize 18 rhabdomyosarcoma and 5 Ewing sarcoma PDX models, not previously characterized. 8. Complete submission of manuscripts reporting PPTC results.